N/A
N=6,718
Transthyretin Amyloidosis Outcome Survey (THAOS)
Transthyretin Gene Mutations · Transthyretin Amyloidosis
Bottom Line
View on ClinicalTrials.gov: NCT00628745 ↗Enrolled (actual)
6,718
Serious AEs
7.7%
Results posted
Nov 2024
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 621; 175; 66; 4 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- None. Observational Study. (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Jun 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
621; 175; 66; 4; 331; 138 | — |
| PRIMARY Number of Participants With Treatment Emergent Treatment Related AEs and SAEs |
47; 11; 1; 2; 28; 5 | — |
| PRIMARY Number of All-Cause Deaths |
158; 1038 | — |
| SECONDARY Number of Participants With Modified Polyneuropathy Disability (mPND) Scores at Baseline |
1860; 512; 1731; 1642; 785; 1372 | — |
| SECONDARY Number of Participants With Coutinho Disease Stages at Baseline |
1860; 512; 1731; 2134; 1040; 1721 | — |
| SECONDARY Number of Participants With Karnofsky Performance Index at Baseline |
2; 1; 2; 6; 0; 6 | — |
| SECONDARY Number of Participants With Heart Failure at Baseline |
2717; 1125; 2245 | — |
| SECONDARY Number of Participants With New York Heart Association (NYHA) Classifications at Baseline |
304; 133; 252; 1341; 557; 1088 | — |
| SECONDARY Number of Participants Diagnosed With ATTR at Baseline |
4773; 2368; 3794; 1234; 36; 1227 | — |
| SECONDARY Number of Participants With Prior Misdiagnosis at Baseline |
616; 283; 493 | — |
| SECONDARY Number of Participants With ATTR Genotypes at Baseline |
4950; 1743; 4365; 1768; 778; 1321 | — |
| SECONDARY Number of Participants With Past or Current Clinical Trial Participation at Baseline |
853; 473; 669; 4417; 1676; 3604 | — |
| SECONDARY Number of Participants With Past or Current Tafamidis Compassionate Use/Early Access or Other Non-commercial Program at Baseline |
85; 80; 38; 3182; 1410; 2423 | — |
| SECONDARY Number of Participants With Known Family History of Symptomatic ATTR at Baseline |
3874; 1365; 3485; 2243; 873; 1792 | — |
| SECONDARY Number of Affected Generations at Baseline |
1.5; 1.5; 1.5 | — |
| SECONDARY Derived Neuropathy Impairment Score-Lower Limb (NIS-LL) at Baseline |
14.0; 20.3; 12.7; 8.2; 7.5; 8.3 | — |
| SECONDARY Body Mass Index (BMI) at Baseline |
26.3; 26.5; 25.9 | — |
| SECONDARY Modified Body Mass Index (mBMI) at Baseline |
1078; 1063.1; 1083.3 | — |
| SECONDARY Sitting Systolic and Diastolic Blood Pressures (SBP and DBP) at Baseline |
123.7; 125.3; 122.9; 75.3; 75.6; 75.1 | — |
| SECONDARY Left Ventricular (LV) Septum Thickness at Baseline |
15.2; 15.3; 15.2 | — |
| SECONDARY Left Ventricular (LV) Ejection Fraction at Baseline |
53.6; 54.3; 53.5 | — |
| SECONDARY Euro Quality of Life-5 Dimensions-3 Level (EQ-5D-3L): Visual Analog Scale (VAS) Overall Health Score at Baseline |
71.0; 70.2; 71.7 | — |
| SECONDARY EQ-5D-3L: VAS Derived Index at Baseline |
0.8; 0.8; 0.8 | — |
| SECONDARY Norfolk Total Quality of Life (QoL) Score at Baseline |
24.3; 25.2; 23.1 | — |
| SECONDARY Number of Participants With Abnormal Electrocardiogram (ECG) at Baseline |
2911; 1226; 2315 | — |
| SECONDARY Number of Participants With Atrial Fibrillation/Flutter, Pacemaker Implanted, and Implantable Cardioverter/Defibrillator (ICD) at Baseline |
667; 290; 501; 364; 167; 266 | — |
Summary
THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate. The principal aims of this outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogenous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.
Eligibility Criteria
Inclusion Criteria: Patients must meet all of the following inclusion criteria to be eligible for inclusion into THAOS:
- Evidence of a personally signed and dated informed consent document indicating that the participant (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
- Males and females greater than or equal to 18 years of age.
- Confirmed genotyped TTR mutation with or without a diagnosis of hereditary or wild-type ATTR amyloidosis. Confirmation of ATTRwt amyloidosis will be determined by genotyped confirmation that patient does not possess a known mutation in TTR gene (ie, is a carrier of wild-type allele only) via genetic testing and one of the following set of criteria (a, b, or c):
- Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
- Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of >12 mm, and presence of amyloid in non-cardiac tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
- Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of >12 mm, and presence of amyloid in cardiac tissue indirectly confirmed by scintigraphy with a "bone seeking tracer" eg, 99mTC-DPD [99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid], 99mTC- PYP [Pyrophosphate], and 99mTC-HMDP [hydroxymethylene diphosphonate] with Perugini grade greater than or equal to 2.
Exclusion Criteria
Patients meeting any of the following will not be included in the study:
- Patient has evidence of primary (light chain) or secondary amyloidosis.
Data sourced from ClinicalTrials.gov (NCT00628745). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.